PRiORiTy Usability Study

Testing an Electronic Patient-Reported Outcome Platform in the Context of Traumatic Brain Injury: PRiORiTy Usability Study

Abstract

Background

Traumatic brain injury (TBI) is a significant public health issue and a leading cause of death and disability globally. Advances in clinical care have improved survival rates, leading to a growing population living with long-term effects of TBI, which can impact physical, cognitive, and emotional health. These effects often require continuous management and individualized care. Traditional paper-based assessments can be cumbersome, potentially impeding regular monitoring of patient-reported outcomes (PROs). Electronic PROs (ePROs) offer a promising alternative by enabling real-time symptom tracking, which can facilitate early identification of issues, support shared decision-making, and improve outcomes for patients with TBI.

Objective

This study evaluates the usability of an ePRO platform—Atom5™—for individuals with TBI. By analyzing how patients use the system to report their symptoms, the study aims to identify usability issues, assess user satisfaction, and determine the potential of Atom5™ to support ongoing patient-centered care.

Methods

Atom5™ was customized to enable individuals with TBI to report their symptoms. Usability testing was conducted through one-on-one sessions with participants recruited from Headway UK—an organization supporting brain injury survivors. Each participant took part in cognitive interviews using with the “Think Aloud” method, encouraging them to verbalize their thoughts and experiences while using the platform. This approach provided qualitative insights into areas of difficulty, usability strengths, and accessibility barriers. User satisfaction was quantitatively assessed with a brief 4-item questionnaire based on the System Usability Scale. Usability outcomes were analyzed for critical and noncritical errors, focusing on user experience and overall satisfaction.

Results

In total, 9 participants completed a single usability testing session using Atom5™, including 4 men, 4 women, and 1 nonbinary individual; 4 participants were under 55 years old, and 6 had their TBI <10 years ago. Finally, 8 participants used an Android device. The platform included measures for anxiety (Generalized Anxiety Disorder-2 item), depression (Patient Health Questionnaire-2), posttraumatic stress disorder (Posttraumatic Stress Disorder checklist 2), and TBI-specific quality of life (Traumatic Brain Injury – Quality of Life Short form) and a total of 26 questions. Overall, all participants were satisfied with the system, noting that it was easy to navigate and accessible despite difficulties in understanding some questions. Further, 6 participants encountered no errors, while 1 participant reported one critical error and 2 others reported one noncritical error each. The participants rated their overall satisfaction with the platform at an average score of 3.9 (SD 0.49) out of 5.

Conclusions

This usability study suggests that individuals living with TBI can effectively report symptoms using the Atom5™ ePRO platform, with generally high satisfaction and few usability issues, thereby enabling continuous monitoring and proactive symptom management. Future ePRO development should focus on inclusivity and adaptability to address the diverse needs of patients with TBI, ensuring these tools can effectively support a wide range of users.


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Lessons Learnt and Share Learnings at the EFGCP Annual Paediatric Conferences

Abstract

For many years, the European Forum for Good Clinical Practice (EFGCP) Children Medicines Working Party has organised a Paediatric conference annually. In the past, this event was organised jointly with the European Medicines Agency who was used to host it, along with the Drug Information Association (DIA). This conference is the opportunity for all involved in paediatric drug development, i.e., regulators, HTA bodies, patients’ representatives, academia and industry, to share learnings and raise awareness about new regulatory requirements of interest to optimise paediatric drug development.

The theme of the 2021 conference was “Challenges and Solutions – the path forward” while in 2022 it focused on “Progress made and Continuing Challenges”. Because of the COVID-19 pandemic these two conferences were organised virtually.

However, this has not impacted the attendance and value of the conference, since because of a broad and attractive agenda there was a wide stakeholder participation, which provided a compendious overview of the leading issues to improve children’s access to innovative medicines.


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A matrix tool to foster patient engagement in children, adolescents and young adults: report from a multistakeholder workshop

Abstract

Background

Patient engagement has become increasingly important in drug development, yet structured guidance to engage children (2-11 years old), adolescents (12-17 years old), and young adults (18-24 years old) is lacking. This study aims to create a matrix tool to foster paediatric patient engagement in drug development by hosting a multistakeholder workshop.

Methods

A matrix tool was co-developed by the study team and the expert group to provide a framework for paediatric patient engagement across drug development stages. The matrix tool’s concept was evaluated using a Delphi-based approach. A pre-workshop questionnaire was sent to 55 experts, followed by an online multistakeholder workshop with 38 participants. A post-workshop questionnaire was redistributed to the initial participants refine the matrix tool further.

Results

The pre-workshop questionnaire (34 respondents) confirmed the need for a co-developed matrix tool, with all statements reaching consensus (cut-off 70% consensus). The workshop (38 participants) revealed strong agreement (92%) on the necessity of such a matrix tool. Participants emphasized the importance of including both chronic and acute diseases, considering patient trial experience, involving families and patient organizations, and aligning age group categories with regulations. The post-workshop questionnaire (18 respondents) showed 89% agreement on involving children, adolescents, and young adults in the matrix tool’s co-development. However, opinions varied on the appropriate minimum age for engagement, ranging from 2 to 17 years.

Conclusions

This study presents the initial steps towards developing a matrix tool to enhance paediatric patient engagement in drug development. The multistakeholder approach provided valuable insights into the matrix tool’s structure and content. While further validation and refinement are needed, particularly regarding practical implementation and global applicability, this initiative lays the groundwork for more effective and inclusive paediatric drug development processes. Future research should focus on validating the matrix tool across different age groups and disease contexts, incorporating real life experiences, and addressing cultural and linguistic considerations.


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Better Medicines for Children: Lessons Learnt and Share Learnings at the EFGCP Annual Paediatric Conferences

Key Themes

1. Tackling Pediatric-Specific Needs in Drug Development

Traditional approaches often do not meet the unique needs of paediatric populations, leading to challenges with dosage and potential side effects when adult drugs are adapted for children. The conferences emphasise the importance of customising research methodologies, clinical trial designs, and formulations to improve safety and efficacy in pediatric treatments.

2. Regulatory Advancements and Challenges

Key discussions addressed regulatory frameworks, including the Paediatric Regulation in the EU and similar initiatives in the U.S. Although these regulations have facilitated paediatric drug development, challenges remain in aligning regulatory requirements with the realities of conducting pediatric trials. Streamlining these processes was a prominent theme, aiming for better regulatory support tailored to paediatric needs.

3. The Power of Collaboration

The conferences showcased how multi-stakeholder collaboration across sectors—industry, academia, government, and patient organisations—drives innovation in pediatric medicine. Success stories highlighted at the events reveal that shared knowledge and resources are instrumental in addressing the logistical and financial challenges that paediatric research often faces.

4. Innovative Clinical Trial Models

With children, clinical trials bring unique ethical and logistical hurdles. Adaptive trials, real-world data, and age-appropriate designs were presented as promising solutions. These approaches balance ethical concerns with scientific rigor, ensuring that paediatric trials yield meaningful data without compromising young patients’ safety.

5. Future Directions and Ongoing Challenges

Topics like adolescent inclusion in adult trials, the role of real-world evidence, and optimising the development process were central to discussions of what comes next. The EU’s ongoing revision of pharmaceutical regulations—including the Paediatric and Orphan Regulations—reflects an evolving commitment to improving access and innovation in paediatric care.


Abstract

For many years, the European Forum for Good Clinical Practice (EFGCP) Children Medicines Working Party has organised a Paediatric conference annually. In the past, this event was organised jointly with the European Medicines Agency who was used to host it, along with the Drug Information Association (DIA). This conference is the opportunity for all involved in paediatric drug development, i.e., regulators, HTA bodies, patients’ representatives, academia and industry, to share learnings and raise awareness about new regulatory requirements of interest to optimise paediatric drug development.

The theme of the 2021 conference was “Challenges and Solutions – the path forward” while in 2022 it focused on “Progress made and Continuing Challenges”. Because of the COVID-19 pandemic these two conferences were organised virtually. However, this has not impacted the attendance and value of the conference, since because of a broad and attractive agenda there was a wide stakeholder participation, which provided a compendious overview of the leading issues to improve children’s access to innovative medicines.


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Using facial reaction analysis and machine learning to objectively assess palatability of medicines in children

For orally administered drugs, palatability is key in ensuring patient acceptability and treatment compliance. Therefore, understanding children’s taste sensitivity and preferences can support formulators in making paediatric medicines more acceptable.

Presently, we explore if the application of computer-vision techniques to videos of children’s reaction to gustatory taste strips can provide an objective assessment of palatability.

Primary school children tasted four different flavoured strips: no taste, bitter, sweet and sour. Data was collected at home, under the supervision of a guardian, with responses recorded using the Aparito Atom5™ app and smartphone camera.

Participants scored each strip on a 5-point hedonic scale. Facial landmarks were identified in the videos, and quantitative measures such as changes around the eyes, nose and mouth were extracted to train models to classify strip taste and score.

We received 197 videos and 256 self-reported scores from 64 participants. The hedonic scale elicited expected results: children like sweetness, dislike bitterness and have varying opinions for sourness. The findings revealed the complexity and variability of facial reactions and highlighted specific measures, such as eyebrow and mouth corner elevations, as significant indicators of palatability. This study into children’s taste specificities can improve the measurement of paediatric medicine acceptability.

An objective measure of how children feel about the taste of medicines has great potential in helping find the most palatable formulation.

Moreover, collecting data in the home setting allows for natural behaviour, with minimal burden for participants.


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Feasibility of an App-Assisted and Home-Based Video Version of the Timed up and Go Test for Patients with Parkinson’s Disease: vTUG

In this paper, our authors assess the feasibility of a digitized COA designed to video record the Timed up and Go (vTUG) test at home using the Atom5™ eCOA platform.

Background

Parkinson’s Disease (PD) is a progressive neurological disorder. Current therapeutic trials investigate treatments that can potentially modify the disease course. Testing their efficiency requires frequent and precise clinical outcome assessments (COA) of symptoms that remain problematic under symptomatic treatments, such as gait and balance. Home-based examinations may enhance patient compliance and, in addition, produce more reliable results by assessing patients more regularly in their familiar surroundings.

Objective

The objective of this study was to assess the feasibility of a digitized COA designed to video record the Timed up and Go (vTUG) test at home via a study-specific smartphone app for patients with PD.

Method

In this study, 28 patients were recruited and asked to perform at home each week a set of three consecutive vTUG tests over a period of 12 weeks using an app. The videos were subjected to a manual review to ascertain the durations of the individual vTUG phases, as well as to identify any errors or deviations in the setup that might have influenced the result. To evaluate the usability and user-friendliness of the vTUG and app, the System Usability Scale (SUS) and User Experience Questionnaire (UEQ) were administered to patients at the study end.


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Video-based assessments of activities of daily living: generating real-world evidence in pediatric rare diseases

Video-based Clinical Outcome Assessments (vCOAs) are poised to revolutionize the management of pediatric rare diseases by providing a more accurate, engaging, and patient-centered approach to monitoring and evaluating treatment outcomes. These methods will become more integrated and essential in clinical trials and everyday care.

In this paper, our authors explore the advantages of vCOAs, the requirements for their implementation and validation, and what the future may hold for further vCOA development.

Abstract

Introduction

Preserving function and independence to perform activities of daily living (ADL) is critical for patients and carers to manage the burden of care and improve quality of life. In children living with rare diseases, video recording ADLs offer the opportunity to collect the patients’ experience in a real-life setting and accurately reflect treatment effectiveness on outcomes that matter to patients and families.

Areas covered

We reviewed the measurement of ADL in pediatric rare diseases and the use of video to develop at-home electronic clinical outcome assessments (eCOA) by leveraging smartphone apps and artificial intelligence-based analysis. We broadly searched PubMed using Boolean combinations of the following MeSH terms ‘Rare Diseases,’ ‘Quality of Life,’ ‘Activities of Daily Living,’ ‘Child,’ ‘Video Recording,’ ‘Outcome Assessment, Healthcare,’ ‘Intellectual disability,’ and ‘Genetic Diseases, Inborn.’ Non-controlled vocabulary was used to include human pose estimation in movement analysis.

Expert opinion

Broad uptake of video eCOA in drug development is linked to the generation of technical and clinical validation evidence to confidently assess a patient’s functional abilities. Software platforms handling video data must align with quality regulations to ensure data integrity, security, and privacy. Regulatory flexibility and optimized validation processes should facilitate video eCOA to support benefit/risk drug assessment.


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The Impact of Video Electronic Outcome Assessments (vCOAs)

Unlock the power of video assessments in your clinical operations with our comprehensive white paper, “From Clinical Visits to Remote Assessments: The Impact of Video Electronic Outcome Assessments (vCOAs).”

This guide explores the use of video Clinical Outcome Assessments (vCOAs), a transformative subset of eCOAs, offering clinical operations teams the tools to capture real-world data more effectively.


In this white paper, you will learn

  1. How vCOAs leverage patients’ own devices to perform tasks and assessments, enhancing data accuracy and reducing biases with automated data analyses.
  2. The benefits of remote video assessments, making trials more inclusive and reducing the need for patients to travel, minimising stress and increasing participation.
  3. The capabilities of real-time tracking and AI-driven analytics in providing immediate, actionable insights, improving the quality and effectiveness of clinical studies.

Three Reasons to Download this White Paper

1. Innovative Trial Design: Gain insights into how vCOAs can invigorate clinical trial designs by enabling remote video assessments and incorporating real-time data analysis.

2. Operational Efficiency: Discover how the automation of vCOAs reduces the burden on clinical staff, streamlines quality control, and ensures high standards of data privacy and security.

3. Improved Patient Outcomes: Learn how continuous and precise monitoring through vCOAs can enhance patient safety, improve treatment outcomes, and provide a more comprehensive understanding of patient health during trials.

Download our white paper and empower your clinical operations team with the knowledge to implement effective, scalable, and patient-centric video eCOAs.

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How to Meet Regulatory & Compliance Requirements for Capturing eCOAs in Clinical Trials

How to Meet Regulatory & Compliance Requirements for Capturing eCOAs in Clinical Trials provides in-depth insights into adapting to the evolving landscape of clinical trials technology.

We highlight the integration of Electronic Data Capture (EDC) platforms, Machine Learning, and AI in clinical management to ensure audit readiness and compliance with the latest FDA and EMA guidelines. Explore the transition from traditional Computer System Validation (CSV) to a more dynamic Computer System Assurance (CSA), focusing on risk-based approaches and minimizing extensive documentation.

In this white paper you will learn:

  1. Transitioning from CSV to CSA. Understand the shift from traditional CSV to the more dynamic CSA. Learn about the FDA’s latest guidelines which emphasize risk-based approaches, reduce the burden of documentation, and ensure the security, availability, and reliability of clinical trial systems.
  2. Adapting to New Regulatory Requirements. Discover how recent regulatory changes impact the management and execution of clinical trials, specifically the integration of Machine Learning and Artificial Intelligence in clinical trial management, and how these technologies meet FDA and EMA standards for data integrity and reliability.
  3. Implementing a Robust eQMS with AMP™. Explore how the Aparito Management Platform (AMP™) streamlines your operations, integrates risk management and ensures data integrity across every aspect of your study, keeping you audit-ready and compliant in a complex regulatory environment.

Download the white paper today to ensure your team is equipped with the knowledge to implement effective and compliant eCOA strategies in your clinical trials. Transform your processes, meet regulatory demands, and stay ahead in the fast-evolving world of clinical research.

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On-demand webinar: Why vCOAs are the Greatest Value Unlock in Clinical Trials

Our webinar on the transformative potential of Video Clinical Outcome Assessments (vCOA) in real-world studies is now available for on-demand viewing!

This webinar is a must-watch for clinicians, researchers, and healthcare professionals seeking to leverage the latest advancements in digital health technology to improve patient outcomes and deepen their understanding of disease progression through innovative assessment tools.

🔍 Discover the Future of Patient Data Capture with vCOA

The webinar offers an in-depth exploration of how vCOA transforms the way patient data is captured, ensuring consistency across remote and in-person assessments. We spotlight the development and validation of the video Timed Up-and-Go (vTUG) assessment within our Atom5™ eCOA platform, a collaboration with DZNE, highlighting its pivotal role in enhancing patient care and research methodologies.

💡 What You Will Learn

1. Validation of Video-Based Outcome Assessments 

Gain insights into our rigorous process for validating the usability and feasibility of video-based assessments, ensuring they meet high standards of accuracy and reliability.

2. Real-World Deployment of vTUG for Parkinson’s Disease

Discover how integrating vTUG into everyday practice is transforming the follow-up of patients with Parkinson’s Disease, enabling continuous, real-world monitoring that captures the nuances of disease progression.

3. Automated Video Analysis for Enhanced Clinical Insights

Learn about the cutting-edge automated analysis of video recordings, which provides more reliable and objective measurements. This technology assists clinicians by introducing new metrics for evaluation and offers unprecedented insight into the progression of diseases.

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